View Cancer Clinical Trials

FHCRC 2007
|
11-697

(Pediatric) Hematopoietic cell transplantation for treatment of patients with primary immunodeficiencies and other nonmalignant inherited disorders using low-dose TBI and fludarabine with or without Campath

Disease(s)

Blood & Marrow Transplant (BMT)

Nonmalignant Inherited Disorders

Primary Immunodeficiencies

Hospital(s)

Main Campus

Phase(s)

N/A

Stage(s)

N/A

Type(s)

Therapeutic

Drug(s)

Alemtuzumab

Fludarabine

Contact Information

Cancer Answer Line

866.223.8100

8:00 am - 4:30 pm, Monday - Friday

Description

Primary

Improve donor chimerism levels in patients with inherited nonmalignant disorders undergoing HCT using a reduced intensity conditioning regimen either through the addition of Campath? or a slightly higher dose of TBI.

Secondary

Decrease the incidence and severity of acute and chronic GVHD through use of marrow as the stem cell source and Campath

Assess disease response following HCT

Immune reconstitution following HCT

Incidence of infections

Overall survival

Percent of patients with CD33/CD19 donor chimerism > 50%

Inclusion Criteria

Age < 55 years with primary immunodeficiency disorder or other nonmalignant inherited disease (except aplastic anemia and fanconi anemia) treatable by allogeneic HCT.

Patients with pre-existing medical conditions or other factors that renders them at high risk for regimen related toxicity or ineligible for a conventional myeloablative HCT.

Hepatic: Patients with clinical or laboratory evidence of liver disease will need to be evaluated for the cause of the liver disease, its clinical severity in terms of liver function and the degree of portal hypertension. Patients will be excluded if they are found to have: fulminant liver failure, cirrhosis of the liver with evidence of portal hypertension, bridging fibrosis, alcoholic hepatitis, esophageal varices, a history of bleeding esophageal varices, hepatic encephalopathy, uncorrectable hepatic synthetic dysfunction evidenced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal liver abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin > 3 mg/dl, or symptomatic biliary disease. Note: Patients will be allowed on to the protocol with liver problems if gastroenterology approves the patient for HCT.

Patients who are seropositive for human immunodeficiency virus (HIV).

Females who are pregnant or breast-feeding.

Fertile men or women who are unwilling to use contraceptives during HCT and up to 12 months post-treatment.

Patients with fungal pneumonia with radiological progression after receipt of amphotericin formulation or mold-active azoles for greater than 1 month will not be eligible for this protocol (either regimen A or B).

Cancer Answers & Appointments

Speak with a cancer nurse specialist for appointment assistance and for answers to your questions about cancer locally at 216.444.7923 or toll-free 1.866.223.8100.

Monday through Friday from 8 a.m. – 4:30 p.m. (ET).

Referrals

Resources for medical professionals

Outpatient appointment referrals: 216.444.7923 or 866.223.8100

Inpatient hospital transfers: 800.553.5056

Referring Physician Concierge: 216.444.6196 or 216.312.4910.

Clinical Trials

This information is provided by Cleveland Clinic and is not intended to replace
the medical advice of your doctor or health care provider.
Please consult your health care provider for advice about a specific medical condition.